Rationally designed ribozymes switched-on by nucleotide repeat extensions as potential tools of genetic therapy for repeat expansion disorders.
Rationally designed ribozymes switched-on by nucleotide repeat extensions as potential tools of genetic therapy for repeat expansion disorders.
批准号:
472596
负责人:
Rouleau Guy A
金额:
$7.29万
依托单位:
依托单位国家:
加拿大
项目类别:
Operating Grants
财政年份:
2022
资助国家:
加拿大
项目状态:
已结题
起止时间:
2022-09-01 至 2023-09-01
中文摘要
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英文摘要
Over 40 diseases, including incurable and fatal neurodegenerative diseases such as ALS, are caused by the expansion of repetitive sequences in the genome. Beyond a certain size, these expansions cause problems in the cells and the body, leading to disease
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Rationally designed ribozymes switched-on by nucleotide repeat extensions as potential tools of genetic therapy for repeat expansion disorders.
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Rationally designed ribozymes switched-on by nucleotide repeat extensions as potential tools of genetic therapy for repeat expansion disorders.
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海外基金